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Table 1 Characteristics of the indications (n = 78)

From: Clinical evidence for orphan medicinal products-a cause for concern?

 

Number of indications (%)

Prevalence of the rare disease in which the indication is authorized

 Between 4/10 000 and 5/10 000

1 (1.3%)

 Between 3/10 000 and 4/10 000

3 (3.8%)

 Between 2/10 000 and 3/10 000

5 (6.4%)

 Between 1/10 000 and 2/10 000

14 (17.9%)

 Between 1/10 000 and 2/10 000

42 (53.8%)

Requested protocol assistance

 Yes

22 (28.2%)

 No

15 (19.2%)

 Not reported

41 (52.6%)

Performed dose finding study

 Yes

44 (56.4%)

 No

25 (32.0%)

 Not reported

9 (11.5%)